results of the RESTORE 1 and 2 clinical trials 2025-09-26 12:32:08

results of the RESTORE 1 and 2 clinical trials

Below is a comprehensive analysis of the results from the RESTORE 1 and RESTORE 2 clinical trials. As there are multiple studies with similar acronyms across different medical fields, this response will focus on the principal RESTORE 1 and 2 trials as identified in the provided texts, covering: (A) RESTORE studies in Type 1 Diabetes (RESTORE-1); (B) RESTORE-IMI 2 in hospital-acquired/ventilator-associated bacterial pneumonia; and (C) RESTORE in esophagogastric cancer survivorship (ReStOre I and II). Each section will address study design, key results, and clinical implications, referencing the supplied sources directly.

A. RESTORE-1 Study: Basal Insulin in Type 1 Diabetes

Study Overview

The RESTORE-1 study was a retrospective, multicenter, non-inferiority study comparing the effectiveness and safety of second-generation basal insulins—insulin glargine 300 U/ml (Gla-300) versus degludec 100 U/ml (IDeg-100)—in adults with type 1 diabetes (T1D) who switched from first-generation basal insulins (1BI). The study utilized electronic medical records and 1:1 propensity score matching for baseline characteristics, with 585 patients in each group. The primary endpoint was change in HbA1c over 6 months, with secondary endpoints including fasting plasma glucose (FPG), body weight, insulin doses, and rates of hypoglycemic events (Laviola et al., 2021).

Key Results

Glycemic Control:
Both groups experienced significant reductions in HbA1c at 6 months:
- Gla-300: Mean reduction of −0.20% (95% CI: −0.32, −0.08)
- IDeg-100: Mean reduction of −0.14% (95% CI: −0.24, −0.04)
Non-inferiority of Gla-300 versus IDeg-100 was confirmed (non-inferiority margin of 0.30%; upper 95% CI at 6 months, 0.09%).
Additional Outcomes:
No significant differences in FPG or body weight between the groups.
Dose changes of basal and short-acting insulin were small, with slightly higher dose increases in the Gla-300 arm (p < 0.006).
Hypoglycemia:
Incidence rates of hypoglycemia (≤70 mg/dl and <54 mg/dl) were slightly lower in the Gla-300 group, though differences were not statistically significant:
- IR ratio for ≤70 mg/dl: 0.82 (95% CI: 0.55, 1.22)
- IR ratio for <54 mg/dl: 0.83 (95% CI: 0.38, 1.83)

Clinical Implications

The RESTORE-1 study demonstrates that both Gla-300 and IDeg-100 are effective and safe options for patients with T1D, with similar efficacy in glycemic control and no significant differences in hypoglycemia risk or weight change after switching from first-generation basal insulins (Laviola et al., 2021).

B. RESTORE-IMI 2 Trial: Imipenem/Relebactam for Hospital-Acquired and Ventilator-Associated Bacterial Pneumonia

Study Overview

RESTORE-IMI 2 was a randomized, controlled, phase 3 clinical trial comparing imipenem/cilastatin/relebactam (IMI/REL) to piperacillin/tazobactam for the treatment of hospital-acquired bacterial pneumonia (HABP) and ventilator-associated bacterial pneumonia (VABP) (Martin-Loeches et al., 2023).

Key Results

A post hoc multivariable regression analysis identified several independent predictors of clinical outcomes:

Day 28 All-Cause Mortality (ACM):
Higher risk associated with vasopressor use, renal impairment, bacteremia at baseline, and APACHE II scores ≥15.
Favorable Clinical Response at Early Follow-Up:
More likely with normal renal function, APACHE II <15, no vasopressor use, and absence of baseline bacteremia.
Favorable Microbiologic Response at End of Treatment:
More likely with IMI/REL treatment, normal renal function, no vasopressor use, nonventilated pneumonia at baseline, ICU admission at randomization, monomicrobial infection, and absence of Acinetobacter calcoaceticus-baumannii complex.
Noninferiority:
The analysis confirmed the noninferiority of IMI/REL to piperacillin/tazobactam for both clinical and microbiological outcomes.

Clinical Implications

RESTORE-IMI 2 validated several well-known predictors of outcomes in severe pneumonia and supported IMI/REL as a noninferior alternative to piperacillin/tazobactam, especially for pathogen eradication. This supports its use as an effective therapy in managing HABP/VABP (Martin-Loeches et al., 2023).

C. ReStOre I & II: Rehabilitation in Esophagogastric and Upper GI Cancer Survivorship

ReStOre I: Pilot RCT

Study Overview

The RESTORE I trial was a randomized controlled trial evaluating a 12-week multidisciplinary rehabilitation program (exercise training, dietary counseling, and education) in disease-free patients after esophagogastric cancer surgery (O'Neill et al., 2018).

Key Results

Cardiorespiratory Fitness (Primary Outcome):
At post-intervention (T1):
- Intervention: 22.20 (SD 4.35) mL·min·kg
- Control: 21.41 (SD 4.49) mL·min·kg
- p < 0.001
At 3-month follow-up (T2):
- Intervention: 21.75 (SD 4.27) mL·min·kg
- Control: 20.74 (SD 4.65) mL·min·kg
- p = 0.001
Secondary Outcomes:
No significant changes in body composition or health-related quality of life (HRQOL).
Adherence:
Mean adherence rates were 94% (supervised) and 78% (unsupervised) in the intervention group.
Safety:
No adverse events reported.

Conclusion

The RESTORE program significantly improved cardiorespiratory fitness in survivors of esophagogastric cancer without compromising body composition, providing proof of principle for rehabilitation in this population (O'Neill et al., 2018).

ReStOre II: Protocol for Larger RCT

Study Overview

RESTORE II is an ongoing randomized controlled trial designed to definitively assess the efficacy of a similar 12-week multidisciplinary rehabilitation program, now extended to a broader cohort including survivors of upper gastrointestinal (UGI) and hepatopancreaticobiliary (HPB) cancers. The protocol specifies a sample size of 120 patients, with primary outcome being cardiorespiratory fitness and secondary outcomes including physical function, body composition, nutritional status, HRQOL, fatigue, and cost-effectiveness (O’Neill et al., 2020).

Key Features

Primary Outcome:
Mean increase in cardiorespiratory fitness (VO2peak) of 3.5 mL/min/kg aimed, with 90% power and 5% significance (allowing for 20% dropout).
Secondary Outcomes:
Physical function, body composition, nutritional status, HRQOL, fatigue.
Design:
Parallel-group, mixed-methods, with both quantitative (objective and subjective measures) and qualitative (focus groups, interviews) components.
Cost-effectiveness and biobanking included.

Status and Implications

As of the latest update, RESTORE II was set to begin in 2020 and aims to provide definitive evidence for multidisciplinary rehabilitation in UGI and HPB cancer survivorship, potentially establishing a model for future clinical care (O’Neill et al., 2020).

Summary Table

| Study | Population | Intervention | Primary Outcome | Key Results/Findings |
|------------------|--------------------------|--------------------------------|----------------------|---------------------------------------------------------------------------------------------------------|
| RESTORE-1 (T1D) | Adults with T1D | Gla-300 vs. IDeg-100 | HbA1c at 6 months | Both insulins non-inferior; similar efficacy and safety (Laviola et al., 2021) |
| RESTORE-IMI 2 | HABP/VABP patients | IMI/REL vs. pip/tazo | Day 28 ACM, response | IMI/REL noninferior; outcome predictors identified (Martin-Loeches et al., 2023) |
| RESTORE I | Esophagogastric CA | Multidisciplinary rehab | VO2peak | Significant improvement in VO2peak; no change in HRQOL/body comp (O'Neill et al., 2018) |
| RESTORE II | UGI & HPB CA survivors | Multidisciplinary rehab | VO2peak, HRQOL | Underway; designed for definitive evidence (O’Neill et al., 2020) |

Conclusion

The RESTORE-1 and RESTORE-IMI 2 trials have provided valuable evidence supporting (1) the non-inferiority of newer basal insulins in T1D, (2) the efficacy of IMI/REL in severe pneumonia, and (3) the benefit of rehabilitation in esophagogastric and upper GI cancer survivors. Ongoing work with RESTORE II aims to further establish multidisciplinary rehabilitation as standard care in cancer survivorship, with robust methodology and a comprehensive suite of outcomes.

References:
- (Laviola et al., 2021)
- (Martin-Loeches et al., 2023)
- (O'Neill et al., 2018)
- (O’Neill et al., 2020)

If you require focused details on a specific RESTORE trial (e.g., diabetes, infectious disease, or cancer rehabilitation), please clarify for a more tailored analysis.

REFERENCES

No Title Found - last accessed: 2025-09-26

InCarda Therapeutics Announces Results From the RESTORE-1 Phase 3 Trial and Plans for Continued Development - Neuro Central - last accessed: 2025-09-26

Comparative Effectiveness of Switching From First-Generation Basal Insulin to Glargine 300 U/ml or Degludec 100 U/ml in Type 1 Diabetes: The RESTORE-1 Study - PubMed - last accessed: 2025-09-26

ClinicalTrials.gov - last accessed: 2025-09-26

Participant- and Disease-Related Factors as Independent Predictors of Treatment Outcomes in the RESTORE-IMI 2 Clinical Trial: A Multivariable Regression Analysis - PubMed - last accessed: 2025-09-26

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC - last accessed: 2025-09-26

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text - last accessed: 2025-09-26

The RESTORE Randomized Controlled Trial: Impact of a Multidisciplinary Rehabilitative Program on Cardiorespiratory Fitness in Esophagogastric cancer Survivorship - PubMed - last accessed: 2025-09-26

Sources used

QUERY: RESTORE 1 AND clinical trial AND results

Direct composite resin fillings versus amalgam fillings for permanent posterior teeth.
The Cochrane database of systematic reviews. 2021/8/13;
DOI: 10.1002/14651858.CD005620.pub3
PMID: 34387873
Abstract
Traditionally, amalgam has been used for filling cavities in posterior teeth, and it continues to be the restorative material of choice in some low- and middle-income countries due to its effectiveness and relatively low cost. However, there are concerns over the use of amalgam restorations (fillings) with regard to mercury release in the body and the environmental impact of mercury disposal. Dental composite resin materials are an aesthetic alternative to amalgam, and their mechanical properties have developed sufficiently to make them suitable for restoring posterior teeth. Nevertheless, composite resin materials may have potential for toxicity to human health and the environment. The United Nations Environment Programme has established the Minamata Convention on Mercury, which is an international treaty that aims "to protect the [sic] human health and the environment from anthropogenic emissions and releases of mercury and mercury compounds". It entered into force in August 2017, and as of February 2021 had been ratified by 127 governments. Ratification involves committing to the adoption of at least two of nine proposed measures to phase down the use of mercury, including amalgam in dentistry. In light of this, we have updated a review originally published in 2014, expanding the scope of the review by undertaking an additional search for harms outcomes. Our review synthesises the results of studies that evaluate the long-term effectiveness and safety of amalgam versus composite resin restorations, and evaluates the level of certainty we can have in that evidence.
To examine the effects (i.e. efficacy and safety) of direct composite resin fillings versus amalgam fillings.
An information specialist searched five bibliographic databases up to 16 February 2021 and used additional search methods to identify published, unpublished and ongoing studies SELECTION CRITERIA: To assess efficacy, we included randomised controlled trials (RCTs) comparing dental composite resin with amalgam restorations in permanent posterior teeth that assessed restoration failure or survival at follow-up of at least three years. To assess safety, we sought non-randomised studies in addition to RCTs that directly compared composite resin and amalgam restorative materials and measured toxicity, sensitivity, allergy, or injury.
We used standard methodological procedures expected by Cochrane.
We included a total of eight studies in this updated review, all of which were RCTs. Two studies used a parallel-group design, and six used a split-mouth design. We judged all of the included studies to be at high risk of bias due to lack of blinding and issues related to unit of analysis. We identified one new trial since the previous version of this review (2014), as well as eight additional papers that assessed safety, all of which related to the two parallel-group studies that were already included in the review. For our primary meta-analyses, we combined data from the two parallel-group trials, which involved 1645 composite restorations and 1365 amalgam restorations in 921 children. We found low-certainty evidence that composite resin restorations had almost double the risk of failure compared to amalgam restorations (risk ratio (RR) 1.89, 95% confidence interval (CI) 1.52 to 2.35; P < 0.001), and were at much higher risk of secondary caries (RR 2.14, 95% CI 1.67 to 2.74; P < 0.001). We found low-certainty evidence that composite resin restorations were not more likely to result in restoration fracture (RR 0.87, 95% CI 0.46 to 1.64; P = 0.66). Six trials used a split-mouth design. We considered these studies separately, as their reliability was compromised due to poor reporting, unit of analysis errors, and variability in methods and findings. Subgroup analysis showed that the findings were consistent with the results of the parallel-group studies. Three trials investigated possible harms of dental restorations. Higher urinary mercury levels were reported amongst children with amalgam restorations in two trials, but the levels were lower than what is known to be toxic. Some differences between amalgam and composite resin groups were observed on certain measures of renal, neuropsychological, and psychosocial function, physical development, and postoperative sensitivity; however, no consistent or clinically important harms were found. We considered that the vast number of comparisons made false-positive results likely. There was no evidence of differences between the amalgam and composite resin groups in neurological symptoms, immune function, and urinary porphyrin excretion. The evidence is of very low certainty, with most harms outcomes reported in only one trial.
Low-certainty evidence suggests that composite resin restorations may have almost double the failure rate of amalgam restorations. The risk of restoration fracture does not seem to be higher with composite resin restorations, but there is a much higher risk of developing secondary caries. Very low-certainty evidence suggests that there may be no clinically important differences in the safety profile of amalgam compared with composite resin dental restorations. This review supports the utility of amalgam restorations, and the results may be particularly useful in parts of the world where amalgam is still the material of choice to restore posterior teeth with proximal caries. Of note, however, is that composite resin materials have undergone important improvements in the years since the trials informing the primary analyses for this review were conducted. The global phase-down of dental amalgam via the Minamata Convention on Mercury is an important consideration when deciding between amalgam and composite resin dental materials. The choice of which dental material to use will depend on shared decision-making between dental providers and patients in the clinic setting, and local directives and protocols.

QUERY: RESTORE 2 AND clinical trial AND results

Use of rubber dam versus cotton roll isolation on composite resin restorations' survival in primary molars: 2-year results from a non-inferiority clinical trial.
BMC oral health. 2022/4/8; Impact Factor: 3.01, Quartile: Q1
DOI: 10.1186/s12903-022-02449-y
PMID: 36217147
Abstract
This non-inferiority randomised clinical trial aimed to evaluate the survival of direct bulk fill composite resin restorations in primary molars using different methods of moisture control: rubber dam isolation (RDI-local anaesthesia and rubber dam) and cotton roll isolation (CRI-cotton roll and saliva ejector). Secondary outcomes included baseline and 2-year incremental cost, self-reported child's pain scores and patient behaviour during the restorative procedure.
A total of 174 molars (93 children) with dentine caries lesions were randomly allocated to study groups (RDI or CRI) and restored with bulk fill composite resin by trained operators. Two blinded examiners assessed the restorations for up to 24 months. Wong-baker faces and Frankl's behaviour rating scales were used for accessing the child's pain and behaviour, respectively. The primary outcome (restoration survival) was analysed using the two-sample non-inferiority test for survival data using Cox Regression (non-inferiority/alternative hypothesis HR > 0.85; CI = 90%). Bootstrap Linear regression was used for cost analysis and logistic regression for pain and behaviour analysis (α = 5%).
After 2-years, 157 restorations were evaluated (drop-out = 9.7%). The survival rate was RDI = 60.4% and CRI = 54.3%. The non-inferiority hypothesis was accepted by the Cox Regression analysis (HR = 1.33; 90% CI 0.88-1.99; p = 0.036). RDI was 53% more expensive when compared to the CRI group. No differences were found between the groups regarding pain (p = 0.073) and behaviour (p = 0.788).
Cotton roll isolation proved to be non-inferior when compared to rubber dam for composite restorations longevity in primary molars. Furthermore, the latest presented the disadvantage of higher cost and longer procedure time. Clinical Significance The moisture control method does not influence the longevity of composite restorations in primary molars. Cotton roll isolation proved to be non-inferior to rubber dam isolation and is a viable option for restoring primary molars. Clinical trial registration registered NCT03733522 on 07/11/2018. The present trial was nested within another clinical trial, the CARies DEtection in Children (CARDEC-03-NCT03520309).

QUERY: RESTORE 1 OR RESTORE 2 AND clinical trial AND results

Interventions for treating cavitated or dentine carious lesions.
The Cochrane database of systematic reviews. 2021/7/19;
DOI: 10.1002/14651858.CD013039.pub2
PMID: 34280957
Abstract
Traditionally, cavitated carious lesions and those extending into dentine have been treated by 'complete' removal of carious tissue, i.e. non-selective removal and conventional restoration (CR). Alternative strategies for managing cavitated or dentine carious lesions remove less or none of the carious tissue and include selective carious tissue removal (or selective excavation (SE)), stepwise carious tissue removal (SW), sealing carious lesions using sealant materials, sealing using preformed metal crowns (Hall Technique, HT), and non-restorative cavity control (NRCC).
To determine the comparative effectiveness of interventions (CR, SE, SW, sealing of carious lesions using sealant materials or preformed metal crowns (HT), or NRCC) to treat carious lesions conventionally considered to require restorations (cavitated or micro-cavitated lesions, or occlusal lesions that are clinically non-cavitated but clinically/radiographically extend into dentine) in primary or permanent teeth with vital (sensitive) pulps.
An information specialist searched four bibliographic databases to 21 July 2020 and used additional search methods to identify published, unpublished and ongoing studies. SELECTION CRITERIA: We included randomised clinical trials comparing different levels of carious tissue removal, as listed above, against each other, placebo, or no treatment. Participants had permanent or primary teeth (or both), and vital pulps (i.e. no irreversible pulpitis/pulp necrosis), and carious lesions conventionally considered to need a restoration (i.e. cavitated lesions, or non- or micro-cavitated lesions radiographically extending into dentine). The primary outcome was failure, a composite measure of pulp exposure, endodontic therapy, tooth extraction, and restorative complications (including resealing of sealed lesions).
Pairs of review authors independently screened search results, extracted data, and assessed the risk of bias in the studies and the overall certainty of the evidence using GRADE criteria. We measured treatment effects through analysing dichotomous outcomes (presence/absence of complications) and expressing them as odds ratios (OR) with 95% confidence intervals (CI). For failure in the subgroup of deep lesions, we used network meta-analysis to assess and rank the relative effectiveness of different interventions.
We included 27 studies with 3350 participants and 4195 teeth/lesions, which were conducted in 11 countries and published between 1977 and 2020. Twenty-four studies used a parallel-group design and three were split-mouth. Two studies included adults only, 20 included children/adolescents only and five included both. Ten studies evaluated permanent teeth, 16 evaluated primary teeth and one evaluated both. Three studies treated non-cavitated lesions; 12 treated cavitated, deep lesions, and 12 treated cavitated but not deep lesions or lesions of varying depth. Seventeen studies compared conventional treatment (CR) with a less invasive treatment: SE (8), SW (4), two HT (2), sealing with sealant materials (4) and NRCC (1). Other comparisons were: SE versus HT (2); SE versus SW (4); SE versus sealing with sealant materials (2); sealant materials versus no sealing (2). Follow-up times varied from no follow-up (pulp exposure during treatment) to 120 months, the most common being 12 to 24 months. All studies were at overall high risk of bias. Effect of interventions Sealing using sealants versus other interventions for non-cavitated or cavitated but not deep lesions There was insufficient evidence of a difference between sealing with sealants and CR (OR 5.00, 95% CI 0.51 to 49.27; 1 study, 41 teeth, permanent teeth, cavitated), sealing versus SE (OR 3.11, 95% CI 0.11 to 85.52; 2 studies, 82 primary teeth, cavitated) or sealing versus no treatment (OR 0.05, 95% CI 0.00 to 2.71; 2 studies, 103 permanent teeth, non-cavitated), but we assessed all as very low-certainty evidence. HT, CR, SE, NRCC for cavitated, but not deep lesions in primary teeth The odds of failure may be higher for CR than HT (OR 8.35, 95% CI 3.73 to 18.68; 2 studies, 249 teeth; low-certainty evidence) and lower for HT than NRCC (OR 0.19, 95% CI 0.05 to 0.74; 1 study, 84 teeth, very low-certainty evidence). There was insufficient evidence of a difference between SE versus HT (OR 8.94, 95% CI 0.57 to 139.67; 2 studies, 586 teeth) or CR versus NRCC (OR 1.16, 95% CI 0.50 to 2.71; 1 study, 102 teeth), both very low-certainty evidence. CR, SE, SW for deep lesions The odds of failure were higher for CR than SW in permanent teeth (OR 2.06, 95% CI 1.34 to 3.17; 3 studies, 398 teeth; moderate-certainty evidence), but not primary teeth (OR 2.43, 95% CI 0.65 to 9.12; 1 study, 63 teeth; very low-certainty evidence). The odds of failure may be higher for CR than SE in permanent teeth (OR 11.32, 95% CI 1.97 to 65.02; 2 studies, 179 teeth) and primary teeth (OR 4.43, 95% CI 1.04 to 18.77; 4 studies, 265 teeth), both very low-certainty evidence. Notably, two studies compared CR versus SE in cavitated, but not deep lesions, with insufficient evidence of a difference in outcome (OR 0.62, 95% CI 0.21 to 1.88; 204 teeth; very low-certainty evidence). The odds of failure were higher for SW than SE in permanent teeth (OR 2.25, 95% CI 1.33 to 3.82; 3 studies, 371 teeth; moderate-certainty evidence), but not primary teeth (OR 2.05, 95% CI 0.49 to 8.62; 2 studies, 126 teeth; very low-certainty evidence). For deep lesions, a network meta-analysis showed the probability of failure to be greatest for CR compared with SE, SW and HT.
Compared with CR, there were lower numbers of failures with HT and SE in the primary dentition, and with SE and SW in the permanent dentition. Most studies showed high risk of bias and limited precision of estimates due to small sample size and typically limited numbers of failures, resulting in assessments of low or very low certainty of evidence for most comparisons.

Web Sources

The RESTORE Randomized Controlled Trial: Impact of a Multidisciplinary Rehabilitative Program on Cardiorespiratory Fitness in Esophagogastric cancer Survivorship - PubMed

. Accordingly, compelling rationale exists to target these impairments in recovery. Methods: Disease-free patients treated for esophagogastric cancer were randomized to either usual care or the 12-week RESTORE program (exercise training, dietary counseling, and multidisciplinary education). The primary outcome was cardiopulmonary exercise testing (VO2peak). Secondary outcomes included body composition (bioimpedance analysis), and HRQOL (EORTC-QLQ-C30). Outcomes were assessed at baseline (T0), postintervention (T1), and at 3-month follow-up (T2). Results: Twenty-two participants were randomized to the control group [mean (standard deviation) age 64.14 (10.46) yr, body mass index 25.67 (4.83) kg/m, time postsurgery 33.68 (19.56) mo], and 21 to the intervention group [age 67.19(7.49) yr, body mass index 25.69(4.02) kg/m, time postsurgery 23.52(15.23) mo]. Mean adherence to prescribed exercise sessions were 94(12)% (supervised) and 78(27)% (unsupervised). Correcting for baseline VO2peak, the intervention arm had significantly higher VO2peak at both T1, 22.20 (4.35) versus 21.41 (4.49) mL · min · kg, P < 0.001, and T2, 21.75 (4.27) versus 20.74 (4.65) mL · min · kg, P = 0.001, compared with the control group. Correcting for baseline values, no changes in body composition or HRQOL were observed. Conclusions: The RESTORE program significantly improved cardiorespiratory fitness of disease-free patients after esophagogastric cancer surgery, without compromise to body composition. This randomized controlled trial provides proof of principle for rehabilitation programs in esophagogastric cancer. Clinical trial registration number: NCT03314311 . PubMed Disclaimer Publication types Randomized Controlled Trial Actions Search in PubMed Search in MeSH Add to Search Research Support, Non-U.S. Gov't Actions Search in PubMed Search in MeSH Add to Search MeSH terms Aged Actions Search in PubMed Search in MeSH Add to Search Body Composition Actions Search in PubMed Search in Me

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

VC < 70%). Recruitment and screening Members of the clinical team will determine potential participants from post-operative clinic lists and from hospital databases. Eligibility screening will be then be processed by the research team and research nurses. Written medical clearance from each participant’s treating consultant will be a pre-requisite to trial enrolment. Potential participants will receive a participant information leaflet (PIL) from a member of the study team. Following receipt of the trial PIL potential participants will then be given a one-week reflection period to consider their interest in trial participation. Upon completion of the reflection window, patients will receive a telephone call to establish whether they wish to participate. Those who express an interest in participation will be asked to attend a screening assessment in which they will provide written informed consent and complete baseline measures. This baseline assessment will take place in the Clinical Research Facility (CRF) at St James’s Hospital (SJH). If accrual is lower than anticipated, recruitment may also be expanded by advertising the study through charity partners; the Irish Cancer Society and the Oesophageal Cancer Fund. Randomisation and blinding Following successful completion of baseline assessments, including a CPET, participants will be formally registered on the trial and will be randomised to the ReStOre II programme or the usual care control group. Block randomisation will be performed using a computer-generated randomisation list. Randomisation will be administered independently by the CRF at SJH. Study assessments will be performed by an assessor concealed to allocation. Given the design of the ReStOre II programme, it will not be feasible to blind either the research staff responsible for delivering the ReStOre intervention or trial participants to their allocation. Intervention The ReStOre II intervention will take place in the exercise physiology suite at the CRF at SJH. The programme will follow a modified version of our established protocol from the ReStOre I study [ 22 ]. The ReStOre II programme comprises three elements: supervised and home-based exercise training, individualised dietetic counselling, and multidisciplinary education. The intervention is summarised in Table 1 . In line with the Irish National Cancer Strategy 2017–2026 [ 24 ], key to the delivery of the programme will be an emphasis on self-management. At the start of the programme participants will set personal goals for the programme. Furthermore, each week participants will also set a specific personal goal for the coming week.

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

new participants. Finally, a patient representative will be invited to speak at the education symposium. Study status ReStOre II will begin in summer 2020. Discussion The ReStOre II RCT, will investigate the efficacy of a 12-week multidisciplinary rehabilitation programme in UGI and HPB cancer survivorship. Whilst the initially feasibility of the programme is established in oesophagogastric cancer survivorship [ 21 , 22 ], ReStOre II will now examine the programme as a definitive intervention in a wider cohort of UGI and HPB cancer survivors. The importance and novel nature of this body of work cannot be understated, particularly in view of the lack of evidence supporting rehabilitative strategies in UGI and HPB survivorship [ 2 ]. ReStOre II will be the first study to examine by RCT the impact of multidisciplinary rehabilitation following surgical resection for pancreatic or hepatocellular carcinoma. ReStOre II will include a 1-year follow-up evaluation of HRQOL, making it the first RCT to examine the longer-term impact of rehabilitation in UGI and HPB cancer survivorship. ReStOre II will also be the first to examine the cost-effectiveness of rehabilitation in UGI and HPB cancer survivorship. Furthermore, ReStOre II will establish a national UGI and HPB cancer survivorship biobank, allowing for innovative investigation of the underpinning biological effects of rehabilitation in the future. A key strength of ReStOre II is the inclusion of cardiorespiratory fitness as the primary outcome. Cardiorespiratory fitness is a key predictor of health, cardiovascular and all-cause mortality, and HRQOL [ 55 , 56 , 57 ], and increasingly is highlighted as a strong predictor of cancer treatment outcomes [ 58 ], and survival [ 59 , 60 ]. ReStOre II is further strengthened by its mixed methods approach. A key finding of the ReStOre pilot RCT was that there were many physical, mental, and social benefits to participation in the ReStOre programme that were not captured by HRQOL questionnaires, but captured instead by the post-intervention focus groups [ 22 , 23 ]. Resultantly, ReStOre II will not only include post-intervention focus groups, but will also incorporate a 1:1 interview at 3-months post-intervention, to explore the longer-term effects of participation. This qualitative approach will provide substantial insight into the

Participant- and Disease-Related Factors as Independent Predictors of Treatment Outcomes in the RESTORE-IMI 2 Clinical Trial: A Multivariable Regression Analysis - PubMed

Participant- and Disease-Related Factors as Independent Predictors of Treatment Outcomes in the RESTORE-IMI 2 Clinical Trial: A Multivariable Regression Analysis - PubMed This site needs JavaScript to work properly. Please enable it to take advantage of the complete set of features! Clipboard, Search History, and several other advanced features are temporarily unavailable. Skip to main page content An official website of the United States government Here's how you know The .gov means it’s official. Federal government websites often end in .gov or .mil. Before
sharing sensitive information, make sure you’re on a federal
government site. The site is secure. The https:// ensures that you are connecting to the
official website and that any information you provide is encrypted
and transmitted securely. Log in Show account info Close Account Logged in as: username Dashboard Publications Account settings Log out Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation Search: Search Advanced Clipboard User Guide Save Email Send to Clipboard My Bibliography Collections Citation manager Display options Display options Format Abstract PubMed PMID Save citation to file Format: Summary (text) PubMed PMID Abstract (text) CSV Create file Cancel Email citation Email address has not been verified. Go to My NCBI account settings to confirm your email and then refresh this page. To: Subject: Body: Format: Summary Summary (text) Abstract Abstract (text) MeSH and other data Send email Cancel Add to Collections Create a new collection Add to an existing collection Name your collection: Name must be less than 100 characters Choose a collection: Unable to load your collection due to an error Please try again Add Cancel Add to My Bibliography My Bibliography Unable to load your delegates due to an error Please try again Add Cancel Your saved search Name of saved search: Search terms: Test search terms Would you like email updates of new search results? Saved Search Alert Radio Buttons Yes No Email: ( change ) Frequency: Monthly Weekly Daily Which day? The first Sunday The first Monday The first Tuesday The first Wednesday The first Thursday The first Friday The first Saturday The first day The first weekday Which day? Sunday Monday Tuesday Wednesday Thursday Friday Saturday Report format: Summary Summary (text) Abstract Abstract (text

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC

MA 01655, United States. Tel.: +1 508 856 2275; E-mail: fred.anderson@umassmed.edu . Prepublished 2020 Feb 7; Collection date 2020. © 2020 – IOS Press and the authors. All rights reserved This is an open access article distributed under the terms of the Creative Commons Attribution (CC BY 4.0) License . PMC Copyright notice PMCID: PMC7739962 PMID: 32039859 Abstract Background: Dramatic improvements in spinal muscular atrophy (SMA) treatment have changed the prognosis for patients with this disease, leading to important new questions. Gathering representative, real-world data about the long-term efficacy and safety of emerging SMA interventions is essential to document their impact on patients and caregivers. Objectives: This registry will assess outcomes in patients with genetically confirmed SMA and provide information on the effectiveness and long-term safety of approved and emerging treatments. Design and Methods: RESTORE is a prospective, multicenter, multinational observational registry. Patients will be managed according to usual clinical practice. Both newly recruitedSMAtreatment centers and sites involved in existing SMA registries, including iSMAC, Treat-NMD, French SMA Assistance Publique- Hôpitaux de Paris (AP-HP), Cure-SMA, SMArtCARE, will be eligible to participate; de novo ; sites already participating in another registry may be included via consortium agreements. Data from patients enrolled in partnering registries will be shared with the RESTORE Registry and data for newly diagnosed patients will be added upon enrollment. Patients will be enrolled over a 5-year period and followed for 15 years or until death. Assessments will include SMA history and treatment, pulmonary, nutritional, and motor milestones, healthcare resource utilization, work productivity, activity impairment, adverse events, quality of life, caregiver burden, and survival. Status: Recruitment started in September 2018. As of January 3, 2020, 64 patients were enrolled at 25 participating sites. Conclusions: The RESTORE Registry has begun recruiting recently diagnosed patients with genetically confirmed SMA, enabling assessment of both short- and long-term patient outcomes. Keywords: Spinal muscular atrophy, multinational, prospective, registry, outcomes, rare disease, long-term follow-up TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT04174157 INTRODUCTION

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC

, representing a broad range of evolving SMA care practices and outcomes across many countries and health care systems. This should provide a robust characterization of the course of disease for patients with SMA receiving the full range of contemporary and evolving treatments and supportive care. Additional strengths of an observational study design include the option to modify the registry design as standards of care are advanced and experience is gained from the care of patients receiving interventions that greatly extend the lifespans of SMA patients. During the planned 15-year duration of the Registry, additional variables may be added to the data collection forms, leading to important insights into questions that were not anticipated in the original study design. In conclusion, RESTORE is a disease registry aimed at assessing the safety and effectiveness of new and evolving treatments for SMA during a 5-year enrollment period and followed for up to 15 years. This project is being undertaken with an open and collaborative approach, with the aim of combining multinational and multi-institutional resources. In addition to providing information about the long-term efficacy and safety of emerging SMA treatments that promise to revolutionize the course of disease for patients with SMA, RESTORE will also assess pharmacoeconomics (e.g., healthcare resource utilization, caregiver burden) and quality-of-care associated with contemporary and evolving SMA treatments. Thus, RESTORE will provide valuable insights into both the effectiveness and value of SMA therapies, representing an essential resource to inform treatment decisions and improve patient outcomes. ACKNOWLEDGMENTS/sources of support All financial and material support for this research was provided by AveXis, Inc., a Novartis company. As stipulated in the RESTORE bylaws, all publication topics must be authored and approved by the members of the steering committee, analyses will be performed by statisticians employed by the CRO that manages the study data. Bob Rhoades provided editorial support for this manuscript. REFERENCES [1]. Sugarman EA, Nagan N, Zhu H, Akmaev VR, Zhou Z, Rohlfs EM, et al.
Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: Clinical laboratory analysis of >72,400 specimens. Eur J Hum Genet. 2012:20(1):27–32. doi: 10.1038/ejhg.2011.134 [ DOI ] [ PMC free article ] [ PubMed ] [ Google Scholar ] [2]. Calucho

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

Ore II will not only include post-intervention focus groups, but will also incorporate a 1:1 interview at 3-months post-intervention, to explore the longer-term effects of participation. This qualitative approach will provide substantial insight into the impact and acceptability of rehabilitation in UGI and HPB cancer survivorship. Moreover, the ReStOre II programme will also be enhanced by a greater focus on self-management strategies, including personalised goal setting. It is anticipated that this will aid participant’s sense of autonomy over their rehabilitation, and therefore optimise long-term adherence to lifestyle changes. ReStOre II will also benefit from the inclusion of PPI initiatives, especially the guidance of the patient collaborator, keeping the patient’s voice central to the ReStOre II study. In conclusion, the ReStOre II RCT, will provide a model of rehabilitation for survivors of UGI and HPB cancer. This unique project addresses a clear gap in cancer research and will help inform much needed future clinical rehabilitative services for UGI and HPB cancer survivors. Availability of data and materials Not applicable. Abbreviations 1-RM : 1-Repition Maximum ACSM : American College of Sports Medicine BIA : Bioimpedance Analysis COPD : Chronic Obstructive Pulmonary Disease CPET : Cardiopulmonary Exercise Test CRF : Clinical Research Facility ECG : Electrocardiograph HGS : Hand Grip Strength HPB : Hepatopancreaticobiliary HRQOL : Health Related Quality of Life HRR : Heart Rate Reserve IDMC : Independent Data Monitoring Committee PPI : Patient and Public Involvement RCT : Randomised Controlled Trial SJH : St James’s Hospital SPPB : Short Physical Performance Battery SWAT : Study Within a Trial TSC : Trial Steering Committee UGI : Upper Gastrointestinal References Clauss D, Tjaden C, Hackert T, Schneider L, Ulrich CM, Wiskemann J, et al. Cardiorespiratory fitness and muscle strength in pancreatic cancer patients. Support Care Cancer. 2017;25(9):2797–807. Article PubMed Google Scholar O'Neill L, Moran J, Guinan EM, Reynolds JV, Hussey J. Physical decline and its implications in the management of oesophageal and gastric cancer: a systematic review. J Cancer Surviv. 2018;12(4):601–18

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

randomised controlled trial (RCT). ReStOre II will now further investigate the efficacy of that programme as it applies to a wider cohort of UGI and HPB cancer survivors, namely survivors of cancer of the oesophagus, stomach, pancreas, and liver. Methods The ReStOre II RCT will compare a 12-week multidisciplinary rehabilitation programme of supervised and self-managed exercise, dietary counselling, and education to standard survivorship care in a cohort of UGI and HPB cancer survivors who are > 3-months post-oesophagectomy/ gastrectomy/ pancreaticoduodenectomy, or major liver resection. One hundred twenty participants (60 per study arm) will be recruited to establish a mean increase in the primary outcome (cardiorespiratory fitness) of 3.5 ml/min/kg with 90% power, 5% significance allowing for 20% drop out. Study outcomes of physical function, body composition, nutritional status, HRQOL, and fatigue will be measured at baseline (T0), post-intervention (T1), and 3-months follow-up (T2). At 1-year follow-up (T3), HRQOL alone will be measured. The impact of ReStOre II on well-being will be examined qualitatively with focus groups/interviews (T1, T2). Bio-samples will be collected from T0-T2 to establish a national UGI and HPB cancer survivorship biobank. The cost effectiveness of ReStOre II will also be analysed. Discussion This RCT will investigate the efficacy of a 12-week multidisciplinary rehabilitation programme for survivors of UGI and HPB cancer compared to standard survivorship care. If effective, ReStOre II will provide an exemplar model of rehabilitation for UGI and HPB cancer survivors. Trial registration The study is registered with ClinicalTrials.gov , registration number: NCT03958019 , date registered: 21/05/2019 Peer Review reports Background With gradually improving survival rates, optimising the quality of upper gastrointestinal (UGI) and hepatopancreaticobiliary (HPB) cancer survivorship has come to the fore of UGI and HPB cancer research. Indeed, the need for rehabilitative strategies to counteract the multitudinous physical and nutritional side effects of UGI and HPB cancers and their treatments has increasingly been

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC

redefining the natural history of a disease resulting from the availability of disease-modifying therapies [ 25 ]. The RESTORE Registry will assess outcomes of patients with a diagnosis of 5q SMA with biallelic deletion of SMN1 , with the primary objective of assessing contemporary SMA treatments, including effectiveness, short- and long-term safety, and overall patient survival. RESTORE will also assess pharmacoeconomic and quality-of-care outcomes of contemporary and evolving SMA treatments, including healthcare resource utilization and caregiver burden. It was designed to overcome the recognized limitations of single-product registries [ 25 ] including difficulty in combining results for research purposes due to lack of comparability in data collected and limited access to data by academic researchers. MATERIALS AND METHODS Design RESTORE is a prospective, multicenter, multinational, non-interventional observational study governed by an international steering committee of SMA experts who are committed to ensuring the quality of these data and to sharing findings through publication and presentation of Registry data. It is the first global SMA registry, consolidating data from multiple countries, established since the advent of gene therapy for SMA. All patients will be managed at participating clinical sites according to best available practices. Clinical care will not be dictated by a research protocol. No additional visits or investigations will be performed beyond those consistent with normal clinical practice. Patients will be enrolled over a 5-year period and followed for 15 years, or until death. Together with individual SMA treatment centers recruited de novo, participating centers may include those involved in existing and evolving SMA registries, including the International SMA Consortium (iSMAC) [ 24, 26 ], Treat-NMD [ 27– 29 ], the French SMA registry, Cure SMA [ 30 ] and SMArtCARE [ 31 ]. Data from patients already enrolled in partnering registries will be transferred to the Registry database, assuming ethical approval and patient consent are obtained as required. Where inclusion of individual patient data is not feasible, aggregate data may be shared. Data for newly diagnosed patients will be added as they are enrolled by their physicians ( Table 1 ). Motor phenotype data will be collected by trained physical therapists. Avoidance of potential duplication of counting patients among these different registries will be accomplished by assigning each patient a Global Unique Identifier. The RESTORE Registry is sponsored by AveXis (a Novartis company), manufacturer of onasemnogene abeparvovec

The RESTORE Randomized Controlled Trial: Impact of a Multidisciplinary Rehabilitative Program on Cardiorespiratory Fitness in Esophagogastric cancer Survivorship - PubMed

Surgery, Trinity Translational Medicine Institute, Trinity College Dublin and St. James's Hospital, Dublin, Ireland. PMID: 30004915 DOI: 10.1097/SLA.0000000000002895 Item in Clipboard Randomized Controlled Trial The RESTORE Randomized Controlled Trial: Impact of a Multidisciplinary Rehabilitative Program on Cardiorespiratory Fitness in Esophagogastric cancer Survivorship Linda M O'Neill et al. Ann Surg . 2018 Nov . Show details Display options Display options Format Abstract PubMed PMID Ann Surg Actions Search in PubMed Search in NLM Catalog Add to Search . 2018 Nov;268(5):747-755. doi: 10.1097/SLA.0000000000002895. Authors Linda M O'Neill 1 , Emer Guinan 2 , Suzanne L Doyle 3 , Annemarie E Bennett 4 , Conor Murphy 5 , Jessie A Elliott 5 , Jacintha OʼSullivan 6 , John V Reynolds 6 , Juliette Hussey 1 Affiliations 1 Discipline of Physiotherapy, School of Medicine, Trinity College Dublin, Dublin, Ireland. 2 School of Medicine, Trinity College Dublin, Dublin, Ireland. 3 School of Biological Sciences, Dublin Institute of Technology, Dublin, Ireland. 4 Department of Clinical Medicine, Trinity College Dublin, Dublin, Ireland. 5 National Esophageal and Gastric Center, St. James's Hospital and Trinity College Dublin, Dublin, Ireland. 6 Department of Surgery, Trinity Translational Medicine Institute, Trinity College Dublin and St. James's Hospital, Dublin, Ireland. PMID: 30004915 DOI: 10.1097/SLA.0000000000002895 Item in Clipboard Cite Display options Display options Format Abstract PubMed PMID Abstract Objective: The Rehabilitation Strategies in Esophagogastric cancer (RESTORE) randomized controlled trial evaluated the efficacy of a 12-week multidisciplinary program to increase the cardiorespiratory fitness and health-related quality of life (HRQOL) of esophagogastric cancer survivors. Background: Patients following treatment for esophagogastric cancer are at risk of physical deconditioning, nutritional compromise, and sarcopenia. Accordingly, compelling rationale exists to target these impairments in recovery. Methods: Disease-free patients treated for esophagogastric cancer were randomized to either usual care or the 12-week RESTORE program (exercise training, dietary counseling,

InCarda Therapeutics Announces Results From the RESTORE-1 Phase 3 Trial and Plans for Continued Development - Neuro Central

InCarda Therapeutics Announces Results From the RESTORE-1 Phase 3 Trial and Plans for Continued Development - Neuro Central Skip to main content Neuro Central is part of Taylor & Francis Group. Taylor & Francis About us Our journals Our Digital Hubs Taylor & Francis Group is a trading division of Informa that operates through various Informa legal entities including, but not limited to, Informa UK Limited, with the registered address at 5 Howick Place, London, SW1P 1WG, UK. Taylor & Francis Home News Topics Care and patient experience Cell biology, pathology and electrophysiology Cognition and psychiatry Drug development Epilepsy and seizure Genetics and gene therapy Imaging and diagnostics Inflammation, immunology and immune disorders Injury, trauma and infection Movement disorders Neurodegeneration and degenerative disease Neuroregeneration and development Oncology and tumor Pain Sleep Stroke Structure and systems neuroscience Technology and techniques Follow us on social media Multimedia Videos Infographics Podcasts Webinars Follow us on social media Features Our Journals Journal Articles eBooks Opinion Interviews Reports Spotlights In Focus Follow us on social media Industry Latest industry news Industry insights Follow us on social media Conferences Events Calendar Follow us on social media Early Careers Articles & Advice Early Career Expert Panel Follow us on social media Register InCarda Therapeutics Announces Results From the RESTORE-1 Phase 3 Trial and Plans for Continued Development 30 Sep 2024 Latest industry news SAN FRANCISCO–(BUSINESS WIRE)–InCarda Therapeutics, Inc.: Analysis of the results of the RESTORE-1 Phase 3 trial following its premature termination demonstrated statistically significant cardioversion of PAF with orally inhaled flecainide, providing compelling rationale for the continued development of FlecIH-103 in the target population of PAF patients. — The RESTORE-1 trial was stopped prematurely due to lower-than-expected efficacy and plasma flecainide levels during a blinded review of the data — There were no safety concerns associated with the administration of FlecIH-103 A novel drug delivery platform has shown the potential to enable efficient and reliable delivery of flecainide to the heart through the lung, appropriate for both hospital and home use Bridging Phase 1 study data to date establishes a path for future registration trials Today, InCarda, a privately held biopharmaceutical company developing an inhaled flecainide acetate inhalation solution (Fle

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC

in US) Patient/Caregiver Reported •Work Productivity and Activity Impairment Questionnaire SMAv2 Outcomes •Zarit Burden Interview •PedsQL Child report •PedsQL Parent report concerning child Serious Adverse Events and Death •Serious adverse events (and adverse events of special interest): ∘ Start and stop dates •Death: ∘ Date of death ∘ Primary cause of death Open in a new tab Data analysis The analysis population will consist of all patients enrolled. The primary analysis will be a summary of outcomes stratified according to the therapy a patient is receiving at the time of enrollment. Descriptive statistics will be presented for the primary analysis. Consistent with the observational nature of this registry, no formal a priori hypothesis testing will be performed. Continuous variables will be summarized using the number of observations, mean, standard deviation, median, minimum, and maximum. Categorical data will be summarized using counts and percentages. Incidence rates (per 10,000 person-years) and 95% confidence intervals for adverse events of special interest will be calculated. Survival will be evaluated using Kaplan-Meier methods. All rates and confidence intervals for individual responses will be performed on actual data. Missing observations will not be imputed in the analysis of individual questions or items. DISCUSSION RESTORE is a prospective, treatment-independent, collaborative global registry. It will recruit patients from a variety of settings and backgrounds allowing assessment of short- and long-term outcomes representative of the contemporary treatment of patients with a confirmed diagnosis of SMA. To permit evaluation of patients who receive gene therapy with onasemnogene abeparvovec, this Registry will recruit patients who were previously treated in a formal clinical study or EAP/MAP. The primary objective of the Registry is to gather long-term follow-up information on patients’ outcomes that cannot be collected in the time frame of a typical clinical trial. Contemporary follow-ups from EAP programs [ 31, 33, 34 ] or clinical trials [ 15, 17, 18 ] do not exceed 2 years and include only patients treated with a single approach. The potential importance of inclusion of patients receiving multiple therapies is underscored by an example from the treatment of Duchenne Muscular Dystrophy, in which a registry enrolling a patient who received several therapies could permit comparison of the long-term efficacies of deflazacort and prednisone [ 35 ], and

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

] and European Society for Clinical Nutrition and Metabolism (ESPEN) [ 32 ] guidelines for cancer survivors . Multidisciplinary education Education sessions ( n = 7) will be delivered weekly during weeks 1–4 and fortnightly thereafter by a range of members of the multidisciplinary team including a doctor, dietitian, occupational therapist, and physiotherapist. Education topics will include an introduction to the ReStOre II programme and talks on items of pertinence to UGI and HPB cancer survivors including; benefits of physical activity, nutrition, management of ongoing medical issues in survivorship, fatigue management, and mindfulness. Standard care group Participants in the control group will continue to receive standard care. Measures ReStOre II study outcomes are listed in Table 3 . The main assessment battery will be performed at; baseline (T0), post-intervention (T1), and 3-months post intervention (T2). Quality of life will be further assessed at 1-year post intervention (T3). At baseline information regarding socio-demographics will be collected from patient interview and data pertaining to medical history, cancer diagnosis and treatments will be obtained from patient’s medical records. Table 3 ReStOre II study outcomes Full size table Primary outcome - cardiopulmonary fitness In ReStOre II, cardiopulmonary fitness, an important index of health [ 33 ], will be measured as the primary outcome during a maximal CPET. The CPET will be performed under medical supervision, using a ramp cycle ergometer protocol with breath-by-breath analysis (COSMED K4B 2 ). The ramp gradient will be set to 10–25 watts/minute based on a calculation using predicted unloaded VO 2 , predicted VO 2 at peak exercise, height, and age using the following standard equations [ 34 ]. 1. VO 2 unloaded in millilitres/minute (ml/min) = 150 + (6 x weight (kg)) 2. Peak VO 2 in ml/min = (height (cm) –age (years)) × 20(sedentary men) or × 14 (sedentary women) 3. Work rate increment minute/watts = ((peak VO 2 ml/min – VO 2 unloaded ml/min)/100) Prior to test commencement, participants will undertake a 3 min warm-up of unloaded cycling. Breath-

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

currently lacking [ 2 ]. Preliminary work at this centre has established the safety, feasibility and initial efficacy of multidisciplinary rehabilitation in oesophago-gastric cancer survivorship [ 20 , 21 , 22 ]. The ReStOre (Rehabilitation Strategies following Oesophago-gastric Cancer) feasibility study and pilot RCT demonstrated that a 12-week programme of supervised and homebased exercise, 1:1 dietary counselling, and health education could result in clinically significant improvements in cardiorespiratory fitness [ 21 , 22 ], and physical and mental well-being [ 23 ] without compromise to body composition. Thus the ReStOre RCT is the first evidence-based model of rehabilitation in UGI cancer survivorship. The ReStOre II RCT will now further examine the effectiveness of the ReStOre programme by RCT in a larger cohort of UGI and HPB cancer survivors. Methods Study aims The primary aim of this work is to examine if a multidisciplinary cancer rehabilitation programme (ReStOre II), incorporating exercise and diet prescription, designed and tailored for disease-free survivors of UGI and HPB cancers, namely cancer of the oesophagus, stomach, pancreas and liver, can lead to improvements in cardiorespiratory fitness in comparison to standard survivorship care. Secondary aims are; To examine the effect of the ReStOre II programme on physical functioning To determine the impact of the ReStOre II programme on body composition To explore the effect of the ReStOre II programme on dietary quality and nutritional status To examine the early and longer-term effects of the ReStOre II programme on patient reported outcomes including HRQOL, and fatigue To qualitatively examine the effects of the ReStOre II programme on physical, mental, and social well-being To evaluate the cost effectiveness of the ReStOre II programme To establish an UGI cancer survivorship biobank for collaborative translational research studies. Study design Using a convergent parallel mixed-methods study design, ReStOre II will be carried out as a randomised controlled trial with two arms: i) an intervention group offered the 12 week ReStOre II programme in addition to usual care, and ii) a control group receiving usual care. The flow of participants through the study is presented in Fig. 1 . The study will recruit participants from three large teaching hospitals in Dublin, Ireland (St James’s Hospital, St Vincent

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC

important for the most severely affected patients, since they constitute the group for which the extent of medical care is the most important. In patients with less severe disease, such as those who are ambulatory, differences in standards of care are less likely to directly and significantly impact patient outcomes. Another limitation of this Registry is the absence of standard training for neuromuscular therapists, and more generally, the lack of standardization across SMA medical assessment. RESTORE was designed to overcome the recognized limitations of single-product registries [ 25, 42 ] including difficulty in combining results for research purposes due to lack of comparability in data collected and limited access to data by academic researchers. When patients are exposed to multiple treatments, particularly at different clinics, partial data for individual patients may be stored in multiple registries. Consequently, their full experience across treatments cannot be appreciated by studying data in a single-product registry. Comparisons of patient data may be limited due to large differences in population characteristics. Addressing this challenge in RESTORE may present an opportunity to answer critical questions about the importance of variations in patients and systems of care. Because patients will not be randomized, statistics will be limited to comparisons of means, frequencies, and temporal trends. The prognostic factors for key milestones, including sitting for the SMA type 1 patients, or walking for type 2 and type 3 patients, will be investigated by comparing proportions of sitters and walkers according to multiple parameters, including disease duration, number of SMN2 copies, baseline conditions, etc. Our focus will be on assuring the representativeness of patients, completeness of the information collected for each patient, and the validity of study data. Due to uncontrolled variations in scheduling follow-up care, data cannot be collected at fixed intervals. Even if all families are offered similar standards of care, some may refuse all or a portion of this support, and variations in Registry findings will reflect the heterogeneity of care delivered. In addition, some patients may choose to enroll in controlled clinical trials during the follow-up period. Loss to follow up, independent of patient status, represents another challenge for data collection and assessment. Strengths of our design include a focus on enrolling consecutive eligible patients who were genetically diagnosed, representing a broad range of evolving SMA care practices and outcomes across many countries and health care systems. This should provide a robust characterization of the course of disease for patients with SMA receiving the full range of contemporary and evolving treatments and supportive care.

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

be assessed for patients in each arm of the study, and alongside the costs of the intervention a comparison will be made of the total costs and outcomes, to estimated cost-effectiveness ratios. Biosample collection Serum, plasma, and whole blood samples will be collected at T0, T1, and T2 for the purpose of establishing a national UGI cancer survivorship biobank. Samples will be processed and stored at -80 °C at the Trinity Translational Medicine Institute, St James’s Hospital, Dublin 8 for future analyses to explore the mechanistic pathways underpinning the impact of multidisciplinary rehabilitation in survivorship. Intervention Fidelity In line with recent findings by Nilsen et al. [ 45 ], our research group also recognises the need to enhance reporting of adherence in exercise oncology trials. To this end, whilst also reporting on traditional adherence variables in our current research portfolio, we are now also endeavouring to report on additional variables adapted from drug trials in all our exercise oncology trials [ 45 ]. The ReStOre II trial will report on standard variables including supervised session attendance and the completion rate of home-based exercise sessions. Akin to our current PRE-HIIT trial examining high intensity interval training in advance of major thoracic surgery for cancer of the lung or oesophagus [ 46 ], ReStOre II will also include a number of novel adherence variables which are outlined fully in Table 4 . Table 4 Exercise adherence variables Full size table Safety Patient safety will be paramount to the implementation of the ReStOre II trial. Standard safety measures will include; written medical clearance, and successful completion of a medically supervised CPET prior to trial commencement. All trial assessments and supervised exercise sessions will take place in the CRF which is located within the confines of SJH and is covered by their emergency response team. All adverse events will be documented, and serious adverse events will be communicated to the SJH/TUH and SVUH research ethics committees. Weight loss is a particular concern for UGI cancer survivors, and accordingly the study dietitian will monitor weight closely during the ReStOre II programme. Sample size calculation Using data from the ReStOre pilot RCT [ 22 ] and supporting published literature [ 47 ], a sample size of 96 (48 per arm) is needed to detect a mean increase of 3.5 ml/min/kg in the intervention arm, assuming a 1.75

Comparative Effectiveness of Switching From First-Generation Basal Insulin to Glargine 300 U/ml or Degludec 100 U/ml in Type 1 Diabetes: The RESTORE-1 Study - PubMed

r.l., Milan, Italy. 5 CORESEARCH, Center for Outcome Research and Clinical Epidemiology, Pescara, Italy. 6 CORESEARCH, Center for Outcome Research and Clinical Epidemiology, Pescara, Italy. nicolucci@coresearch.it. PMID: 33351177 PMCID: PMC7846660 DOI: 10.1007/s13300-020-00982-z Item in Clipboard Comparative Effectiveness of Switching From First-Generation Basal Insulin to Glargine 300 U/ml or Degludec 100 U/ml in Type 1 Diabetes: The RESTORE-1 Study Luigi Laviola et al. Diabetes Ther . 2021 Feb . Show details Display options Display options Format Abstract PubMed PMID Diabetes Ther Actions Search in PubMed Search in NLM Catalog Add to Search . 2021 Feb;12(2):509-525. doi: 10.1007/s13300-020-00982-z. Epub 2020 Dec 22. Authors Luigi Laviola 1 , Francesca Porcellati 2 , Daniela Bruttomesso 3 , Monica Larosa 4 , Maria Chiara Rossi 5 , Antonio Nicolucci 6 ; RESTORE-1 Study Group Affiliations 1 Endocrinology, Andrology and Metabolic Diseases, University of Bari Aldo Moro, Bari, Italy. 2 Section of Internal Medicine, Endocrinology and Metabolism, Department of Medicine, Perugia University School of Medicine, Perugia, Italy. 3 Department of Medicine, University of Padova, Padua, Italy. 4 Medical Affairs, Sanofi S.r.l., Milan, Italy. 5 CORESEARCH, Center for Outcome Research and Clinical Epidemiology, Pescara, Italy. 6 CORESEARCH, Center for Outcome Research and Clinical Epidemiology, Pescara, Italy. nicolucci@coresearch.it. PMID: 33351177 PMCID: PMC7846660 DOI: 10.1007/s13300-020-00982-z Item in Clipboard Full text links Cite Display options Display options Format Abstract PubMed PMID Abstract Introduction: Following pivotal trials, real-world evidence is important to assess the impact of new drugs in everyday clinical practice. The RESTORE-1 study aimed to compare effectiveness and safety of the second-generation basal insulins (

The RESTORE Randomized Controlled Trial: Impact of a Multidisciplinary Rehabilitative Program on Cardiorespiratory Fitness in Esophagogastric cancer Survivorship - PubMed

The RESTORE Randomized Controlled Trial: Impact of a Multidisciplinary Rehabilitative Program on Cardiorespiratory Fitness in Esophagogastric cancer Survivorship - PubMed This site needs JavaScript to work properly. Please enable it to take advantage of the complete set of features! Clipboard, Search History, and several other advanced features are temporarily unavailable. Skip to main page content An official website of the United States government Here's how you know The .gov means it’s official. Federal government websites often end in .gov or .mil. Before
sharing sensitive information, make sure you’re on a federal
government site. The site is secure. The https:// ensures that you are connecting to the
official website and that any information you provide is encrypted
and transmitted securely. Log in Show account info Close Account Logged in as: username Dashboard Publications Account settings Log out Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation Search: Search Advanced Clipboard User Guide Save Email Send to Clipboard My Bibliography Collections Citation manager Display options Display options Format Abstract PubMed PMID Save citation to file Format: Summary (text) PubMed PMID Abstract (text) CSV Create file Cancel Email citation Email address has not been verified. Go to My NCBI account settings to confirm your email and then refresh this page. To: Subject: Body: Format: Summary Summary (text) Abstract Abstract (text) MeSH and other data Send email Cancel Add to Collections Create a new collection Add to an existing collection Name your collection: Name must be less than 100 characters Choose a collection: Unable to load your collection due to an error Please try again Add Cancel Add to My Bibliography My Bibliography Unable to load your delegates due to an error Please try again Add Cancel Your saved search Name of saved search: Search terms: Test search terms Would you like email updates of new search results? Saved Search Alert Radio Buttons Yes No Email: ( change ) Frequency: Monthly Weekly Daily Which day? The first Sunday The first Monday The first Tuesday The first Wednesday The first Thursday The first Friday The first Saturday The first day The first weekday Which day? Sunday Monday Tuesday Wednesday Thursday Friday Saturday Report format: Summary Summary (text) Abstract Abstract (text

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC

of counting patients among these different registries will be accomplished by assigning each patient a Global Unique Identifier. The RESTORE Registry is sponsored by AveXis (a Novartis company), manufacturer of onasemnogene abeparvovec, a gene therapy for SMA. The registry data are owned by the sponsor, which has developed bylaws providing for control of publications under the authority of a Steering Committee comprised of academic physicians and scientists with expertise in SMA. Table 1. Data sources used for the RESTORE Registry •Individual de novo clinical sites •Existing SMA Consortia •Managed Access Programs •Expanded Access Programs •Post Marketing Surveillance (required follow up) Open in a new tab Ethical considerations RESTORE is being conducted in accordance with established research principles, local treatment practices and regulations, and guidelines of the International Council on Harmonisation. At centers recruited de novo , physicians will be asked to provide documentation and approvals per local regulations. Once a site is activated, physicians will consent patients using an Informed Consent Form (ICF) that has been approved by their Independent Ethics Committee/Institutional Review Board in accordance with local practices and regulations. Prior to any data collection, a written ICF and a privacy statement will be signed by the parent/guardian; and, where appropriate, by the patient. All information obtained during the conduct of the Registry with respect to the patient’s identity or state of health will be treated as confidential. Prior to any disclosure of protected personal information, a signed written agreement will be obtained from the patient or his/her legal representative. Patients All enrolled patients must have 5q SMA diagnosed at <18 years of age that is genetically confirmed (bi-allelic loss or pathological variants of the SMN1 gene) [ 32 ]. Subtypes of SMA will be categorized based on age at onset, number of SMN2 copies, and highest acquired motor function (e.g., independent sitting and ambulation). All patient recruitment and data management will be conducted in accordance with FDA/EMA requirements. During a 5-year enrollment period, RESTORE will document the long-term safety and effectiveness of gene therapy, along with that of evolving and emerging SMA treatments, including up to 15-years of follow-up. To minimize selection bias, consecutive eligible patients will be enrolled from participating clinical sites. To obtain adequate patient numbers to support this new observational study, an international collaboration of existing S

Participant- and Disease-Related Factors as Independent Predictors of Treatment Outcomes in the RESTORE-IMI 2 Clinical Trial: A Multivariable Regression Analysis - PubMed

Louis, Missouri, USA. 4 Merck & Co, Inc, Rahway, New Jersey, USA. PMID: 37383243 PMCID: PMC10297016 DOI: 10.1093/ofid/ofad225 Item in Clipboard Participant- and Disease-Related Factors as Independent Predictors of Treatment Outcomes in the RESTORE-IMI 2 Clinical Trial: A Multivariable Regression Analysis Ignacio Martin-Loeches et al. Open Forum Infect Dis . 2023 . Show details Display options Display options Format Abstract PubMed PMID Open Forum Infect Dis Actions Search in PubMed Search in NLM Catalog Add to Search . 2023 May 4;10(6):ofad225. doi: 10.1093/ofid/ofad225. eCollection 2023 Jun. Authors Ignacio Martin-Loeches 1 , Andrew F Shorr 2 , Marin H Kollef 3 , Jiejun Du 4 , Maria C Losada 4 , Amanda Paschke 4 , C Andrew DeRyke 4 , Michael Wong 4 , Erin H Jensen 4 , Luke F Chen 4 Affiliations 1 Department of Intensive Care Medicine, Multidisciplinary Intensive Care Research Organization, St James's University Hospital, Trinity Centre for Health Sciences, Dublin, Ireland. 2 Section of Pulmonary, Critical Care, and Respiratory Services, MedStar Washington Hospital Center, Washington, District of Columbia, USA. 3 Division of Pulmonary and Critical Care Medicine, Department of Internal Medicine, Washington University School of Medicine, St Louis, Missouri, USA. 4 Merck & Co, Inc, Rahway, New Jersey, USA. PMID: 37383243 PMCID: PMC10297016 DOI: 10.1093/ofid/ofad225 Item in Clipboard Cite Display options Display options Format Abstract PubMed PMID Abstract Background: In the RESTORE-IMI 2 trial, imipenem/cilastatin/relebactam (IMI/REL) was noninferior to piperacillin/tazobactam in treating hospital-acquired bacterial pneumonia/ventilator-associated bacterial pneumonia. This post hoc analysis was conducted to determine independent predictors of efficacy outcomes in the RESTORE-IMI 2 trial, to assist in treatment decision making. Methods: A stepwise multivariable regression analysis was conducted to identify variables that were independently associated with day 28 all-cause mortality

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC

nutritional assessment, motor milestone assessments) and standardized patient and caregiver reported outcomes assessed in the registry are listed in Table 2 . Table 2. Study variables collected in the RESTORE Registry Category Variables Confirmation of Eligibility, •Date of informed consent for study enrollment Socio-demographics, Study Status •Eligibility Assessment •Socio-demographic characteristics: ∘ Year of Birth ∘ Gestational age ∘ Gender ∘ Race ∘ Ethnicity (US) •Withdrawal of consent: ∘ Date of withdrawal ∘ Reason for withdrawal Clinical Characteristics of Patient •Medical history: ∘ History of SMA: ■ Date and age of diagnosis ■ Genetic status ■ SMN2 copy number ■ Point mutation ■ Weight at diagnosis of SMA ■ Length/height at diagnosis of SMA ∘ Other medical history ∘ Family history: ■ Maternal and paternal genetic test results Treatments ■ Onasemnogene abeparvovec treatment (if applicable): ∘ Date of treatment, dose •Prednisolone treatment (onasemnogene abeparvovec group only) •Nusinersen treatment: ∘ Dose and frequency ∘ Start and stop dates •Other concomitant medications: ∘ Dose and frequency ∘ Start and stop dates Patient Assessments •Pulmonary assessments: ∘ Was it performed, normal/abnormal, if abnormal details •Ventilatory support: ∘ Cough assist details ∘ Non-invasive details ∘ Invasive details •Nutritional assessment: ∘ Use of non-oral procedure to administer food: ∘ Details including start/stop, volume, frequency, caloric intake •Motor milestone assessments: ∘ Developmental milestones ∘ Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) ∘ Hammersmith Infant Neurological Examination (HINE) ∘ Hammersmith Functional Motor Scale •Laboratories ∘ Liver function test Hospitalization and Healthcare •Emergency room visits and hospitalizations: Resource Utilization ∘ Date of hospitalizations ∘ Reason of hospitalizations •Other therapies and visits •Insurance type (in US) Patient/Caregiver Reported •Work Productivity and Activity Impairment Questionnaire SMAv2 Outcomes •Zarit Burden Interview •PedsQL Child report •PedsQL Parent report concerning child Serious Adverse

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

24 ], key to the delivery of the programme will be an emphasis on self-management. At the start of the programme participants will set personal goals for the programme. Furthermore, each week participants will also set a specific personal goal for the coming week. Table 1 The ReStOre II programme Full size table Exercise The exercise component will consist of a 12-week supervised and home-based intervention. The exercise prescription will include both aerobic and resistance training and will be prescribed by a physiotherapist. Supervised group exercise sessions will be held twice weekly during the first 4 weeks to reintroduce exercise to participants in a safe and structured manner. As the programme progresses the frequency of supervised sessions will decrease, and the frequency of home-based exercise sessions will increase. This structure aims to encourage self-management in survivorship and increase autonomy with exercise prescription. The ReStOre II exercise prescription is presented in Table 2 . Aerobic training intensity will be individualised to the participant’s fitness. Exercise intensity will be prescribed using heart rate reserve (HRR) calculated using the Karvonen formula (HRR = maximum heart rate –resting heart rate) [ 25 ]. The values for maximum heart rate and resting heart rate will be calculated during the baseline CPET. Participants will wear Polar Heart Rate monitors to ensure compliance with the prescribed exercise intensity. Intensity will also be monitored with the Borg Perceived Scale of Exertion [ 26 ]. Upon completion of the ReStOre II programme, participants will be completing 150 min of moderate-vigorous intensity activity per week, as per ACSM physical activity guidelines [ 16 ]. Resistance training will also be tailored to the participant’s fitness levels. Specifically, the first week will be used to ensure safe and appropriate technique on all exercises and determine the training loads for subsequent sessions. The program will consist of 5 major movements (squat, lunge, hip flexion/extension, pushing and pulling) incorporating compound exercises targeting major muscle groups of the upper and lower body. Additionally, accessory movements targeting the biceps and triceps will be incorporated. Resistance will be added using free weights, resistance bands, a leg press machine, or body weight. Participants will be provided with resistance equipment for use at home. Where possible, loading will be progressed throughout the programme using the “2 for 2 rule” [ 27 ]. If an individual can complete two additional repetitions of an exercise, for 2 consecutive sessions,

The RESTORE Randomized Controlled Trial: Impact of a Multidisciplinary Rehabilitative Program on Cardiorespiratory Fitness in Esophagogastric cancer Survivorship - PubMed

? The first Sunday The first Monday The first Tuesday The first Wednesday The first Thursday The first Friday The first Saturday The first day The first weekday Which day? Sunday Monday Tuesday Wednesday Thursday Friday Saturday Report format: Summary Summary (text) Abstract Abstract (text) PubMed Send at most: 1 item 5 items 10 items 20 items 50 items 100 items 200 items Send even when there aren't any new results Optional text in email: Save Cancel Create a file for external citation management software Create file Cancel Your RSS Feed Name of RSS Feed: Number of items displayed: 5 10 15 20 50 100 Create RSS Cancel RSS Link Copy Actions Cite Collections Add to Collections Create a new collection Add to an existing collection Name your collection: Name must be less than 100 characters Choose a collection: Unable to load your collection due to an error Please try again Add Cancel Permalink Permalink Copy Display options Display options Format Abstract PubMed PMID Page navigation Title & authors Abstract Publication types MeSH terms Associated data Grants and funding LinkOut - more resources Title & authors Abstract Publication types MeSH terms Associated data Grants and funding LinkOut - more resources Randomized Controlled Trial Ann Surg Actions Search in PubMed Search in NLM Catalog Add to Search . 2018 Nov;268(5):747-755. doi: 10.1097/SLA.0000000000002895. The RESTORE Randomized Controlled Trial: Impact of a Multidisciplinary Rehabilitative Program on Cardiorespiratory Fitness in Esophagogastric cancer Survivorship Linda M O'Neill 1 , Emer Guinan 2 , Suzanne L Doyle 3 , Annemarie E Bennett 4 , Conor Murphy 5 , Jessie A Elliott 5 , Jacintha OʼSullivan 6 , John V Reynolds 6 , Juliette Hussey 1 Affiliations Expand Affiliations 1 Discipline of Physiotherapy, School of Medicine, Trinity College Dublin, Dublin, Ireland. 2 School of Medicine, Trinity College Dublin, Dublin, Ireland. 3 School of Biological Sciences, Dublin Institute of Technology, Dublin, Ireland. 4 Department of Clinical Medicine, Trinity College Dublin, Dublin, Ireland. 5 National Esophageal and Gastric Center, St. James's Hospital and Trinity College Dublin, Dublin, Ireland. 6 Department of Surgery, Trinity Translational Medicine Institute, Trinity College Dublin and St. James's Hospital, Dublin, Ireland. PMID: 30004915 DOI: 10.1097/SLA.0000000000002895 Item in Clipboard Randomized Controlled

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC

emerging SMA treatments, including up to 15-years of follow-up. To minimize selection bias, consecutive eligible patients will be enrolled from participating clinical sites. To obtain adequate patient numbers to support this new observational study, an international collaboration of existing SMA registries and de novo clinical sites is being created. Patients will be recruited worldwide to provide sufficient data to document differences between patients who receive a variety of treatments.as they evolve during the 15-year duration of this registry. Registry procedures Patient care will follow usual SMA treatment practices in each country and participating clinical site. No additional diagnostic or monitoring procedures will be required beyond the routine clinical practices at each participating clinic or hospital. Treatment The choice of ongoing medical treatment for the duration of the Registry will be made independently by the physician in the course of regular patient care and will not be influenced by participation in this Registry. Physicians are free to add or withdraw any medication but will continue to monitor each patient for the full 15 years, until death, or until the patient is withdrawn from the Registry, which may occur at the discretion of the patient or the patient’s parent/legal representative or physician. No treatments will be provided by the sponsor as part of participation in this Registry. Data acquisition and variables assessed No mandatory visits, tests, or assessments are required for participation in this Registry. All follow-up visits will be scheduled and conducted according to each participating site’s usual clinical practices. Data acquisition Sources that will generate data for this Registry include consortia, individual clinical sites, managed- and expanded-access Programs (MAP/EAP), and post-marketing study obligations. Additional prospective data for SMA patients that meet RESTORE eligibility criteria may be extracted from existing registries that agree to share their information ( Table 1 ). Additional data will be collected from de novo study sites by local study coordinators who will complete online electronic data capture based on information in the patient’s medical chart. Additionally, for de novo patients, self-reported data will be collected using standardized patient reported outcome questionnaires and caregiver surveys. Study variables (e.g., socio-demographic characteristics, history of SMA, pulmonary assessments, ventilatory support, nutritional assessment, motor milestone assessments) and standardized patient and caregiver reported outcomes assessed in the registry are listed in Table 2 . Table 2. Study variables collected in the RESTORE Registry Category Variables Confirmation of Eligibility, •Date of

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

all patients with analysable data irrespective of fidelity, compliance, or arm cross-over. Statisticians will finalise a detailed Statistical Analysis Plan prior to the final T3 visit taking place and will remain blinded to study arm until the analysis is complete. A qualitative descriptive approach [ 48 ] will be taken to the analysis of qualitative data. Braun and Clarke’s 6 stage approach to thematic analysis will be used to analyse all data collected [ 49 ]. A team of researchers will analyse all transcripts following an agreed process using nVivo 12 (QSR International, Australia). Trial management and governance Management of the ReStOre II study will be overseen by three committees; a Trial management Group (TMG), Trial Steering Committee (TSC) and an Independent Data Monitoring Committee (IDMC). The TMG will oversee the daily trial management. The TSC will meet biannually and provide oversight of the trial and ensure the trial is conducted in accordance with the principles of Good Clinical Practice. The IDMC will monitor trial data to ensure the safety of the participants . The IDMC will meet biannually to review interim safety and accrual data. In addition, the IDMC may also meet at the discretion of the TSC. Dissemination Findings of ReStOre II will be disseminated via peer-reviewed publications and conference presentations. Aggregate study results will be presented to participants and their families at an education symposium upon study completion. Anonymised data and all computer code used for the analyses will be made available on an open access repository. Public and patient involvement (PPI) ReStOre II will involve a number of PPI initiatives. Firstly, a previous ReStOre participant is a collaborator on the trial and will sit on the TSC, and will review the study protocol, study procedures and documentation. Second, we have incorporated a PPI focused study within a trial (SWAT) into this study [ 50 , 51 , 52 , 53 ]. The SWAT will explore the effects of patient co-designed participant information on study recruitment rates. The protocol for the SWAT has been published separately [ 54 ]. Third, past ReStOre participants will attend the first class of each programme to meet and encourage new participants. Finally, a patient representative will be invited to speak at the education symposium. Study status ReStOre II will begin in summer 2020. Discussion The ReStOre II RCT, will investigate the efficacy of a 12-week mult

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

care, and ii) a control group receiving usual care. The flow of participants through the study is presented in Fig. 1 . The study will recruit participants from three large teaching hospitals in Dublin, Ireland (St James’s Hospital, St Vincent’s University Hospital, and Tallaght University Hospital). Ethical approval has been granted from their respective research ethics committees and any subsequent amendments to the trial protocol will be submitted for their approval. The study will be conducted in accordance with the Declaration of Helsinki. Fig. 1 Participant flow through study Full size image Study participants ReStOre II will recruit 120 patients with a histological confirmed diagnosis of cancer of the oesophagus, stomach, pancreas, or liver who have undergone surgery with curative intent. Participants must meet the following eligibility criteria; be ≥ three months post oesophagectomy, total gastrectomy, pancreaticoduodenectomy, or major liver resection, ± neo-adjuvant/adjuvant chemo/chemoradiotherapy with curative intent, and adjuvant therapy must be completed. Exclusion criteria are; ongoing serious post-operative morbidity, and, evidence of active or recurrent disease. In addition, those with any serious co-morbidity that would impact on exercise participation will be excluded, including those with; electrocardiograph (ECG) abnormalities at rest or during Cardiopulmonary Exercise Test (CPET), congestive heart failure (NY Heart Association Class II, III or IV), uncontrolled hypertension (resting systolic blood pressure > 180 mmHg and/or diastolic > 100 mmHg), recent serious cardiovascular events (within 12 months) including, but not limited to, cerebrovascular accident, and myocardial infarction, unstable cardiac, renal, lung, liver or other severe chronic disease, uncontrolled atrial fibrillation, or left ventricular function < 50%, %; and, severe/ very severe chronic obstructive disease (COPD) (GOLD Stages III/IV FEV1 < 50%, FEV1/FVC < 70%). Recruitment and screening Members of the clinical team will determine potential participants from post-operative clinic lists and from hospital databases. Eligibility screening will be then be processed by the research team and research nurses.

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text Skip to main content Advertisement Search Explore journals Get published About BMC My account Search all BMC articles Search BMC Cancer Home About Articles Submission Guidelines Collections Join the Board Submit manuscript Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial Download PDF Download PDF Study protocol Open access Published: 13 May 2020 Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial Linda O’Neill ORCID: orcid.org/0000-0002-0109-9650 1 , Emer Guinan 2 , Suzanne Doyle 3 , Deirdre Connolly 4 , Jacintha O’Sullivan 5 , Annemarie Bennett 6 , Grainne Sheill 1 , Ricardo Segurado 7 , Peter Knapp 8 , Ciaran Fairman 9 , Charles Normand 10 , Justin Geoghegan 11 , Kevin Conlon 11 , 12 , 13 , John V. Reynolds 5 & … Juliette Hussey 1 Show authors BMC Cancer volume 20 , Article number: 415 ( 2020 ) Cite this article 6857 Accesses 23 Citations 4 Altmetric Metrics details Abstract Background Curative treatment for upper gastrointestinal (UGI) and hepatopancreaticobiliary (HPB) cancers, involves complex surgical resection often in combination with neoadjuvant/adjuvant chemo/chemoradiotherapy. With advancing survival rates, there is an emergent cohort of UGI and HPB cancer survivors with physical and nutritional deficits, resultant from both the cancer and its treatments. Therefore, rehabilitation to counteract these impairments is required to maximise health related quality of life (HRQOL) in survivorship. The initial feasibility of a multidisciplinary rehabilitation programme for UGI survivors was established in the Rehabilitation Strategies following Oesophago-gastric Cancer (ReStOre) feasibility study and pilot randomised controlled trial (RCT). ReStOre II will now further investigate the efficacy of that programme as it applies to a wider cohort of UGI and HPB cancer survivors, namely survivors of cancer of the oesophagus, stomach,

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC Skip to main content An official website of the United States government Here's how you know Here's how you know Official websites use .gov A .gov website belongs to an official
government organization in the United States. Secure .gov websites use HTTPS A lock ( Lock Locked padlock icon ) or https:// means you've safely
connected to the .gov website. Share sensitive
information only on official, secure websites. Search Log in Dashboard Publications Account settings Log out Search… Search NCBI Primary site navigation Search Logged in as: Dashboard Publications Account settings Log in Search PMC Full-Text Archive Search in PMC Journal List User Guide PERMALINK Copy As a library, NLM provides access to scientific literature. Inclusion in an NLM database does not imply endorsement of, or agreement with,
the contents by NLM or the National Institutes of Health. Learn more: PMC Disclaimer | PMC Copyright Notice J Neuromuscul Dis . 2020 Mar 20;7(2):145–152. doi: 10.3233/JND-190451 Search in PMC Search in PubMed View in NLM Catalog Add to search RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design Richard S Finkel Richard S Finkel a Department of Pediatrics, Division of Neurology, Nemours Children’s Hospital, Orlando, FL, United States Find articles by Richard S Finkel a , John W Day John W Day b Department of Neurology, Stanford University Medical Center, Stanford, CA, United States Find articles by John W Day b , Darryl C De Vivo Darryl C De Vivo c Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, NY, United States Find articles by D

Comparative Effectiveness of Switching From First-Generation Basal Insulin to Glargine 300 U/ml or Degludec 100 U/ml in Type 1 Diabetes: The RESTORE-1 Study - PubMed

PubMed PMID Abstract Introduction: Following pivotal trials, real-world evidence is important to assess the impact of new drugs in everyday clinical practice. The RESTORE-1 study aimed to compare effectiveness and safety of the second-generation basal insulins (2BI), i.e., insulin glargine 300 U/ml (Gla-300) vs. degludec 100 U/ml (IDeg-100), in type 1 diabetes (T1D). Methods: Retrospective, non-inferiority, multicenter study, based on electronic medical records. All patients switching to Gla-300 or IDeg-100 from first-generation basal insulins (1BI) were 1:1 propensity score matched (PSM). Changes during 6 months in HbA1c (primary endpoint), fasting plasma glucose (FPG), body weight, and insulin doses were assessed using linear mixed models for repeated measures. Incidence rates (IR) of hypoglycemic events were assessed. Results: Overall, 19 centers provided data on 585 patients in each PSM cohort. For both groups, statistically significant reductions in HbA1c from baseline to 6 months were documented: - 0.20%; (95% CI - 0.32; - 0.08) in the Gla-300 group and - 0.14%; (95% CI - 0.24; - 0.04) in the IDeg-100 group. The non-inferiority of Gla-300 vs. IDeg-100 was confirmed (non-inferiority margin of 0.30%; upper 95% CI at 6 months, 0.09%). No statistically significant between-group differences emerged in FPG and body weight. Dose changes of basal and short-acting insulin were small in both groups, but higher in the Gla-300 group than in the Deg-100 group (p < 0.006). Incidence rates (IR) of hypoglycemia (blood glucose ≤ 70 mg/dl and < 54 mg/dl) during the 6-month follow-up by treatment were slightly lower in the Gla-300 group than in the Deg-100 group [IR ratios 0.82 (95% CI 0.55; 1.22) and 0.83; (95% CI 0.38; 1.83), respectively]. Hypoglycemic events (blood glucose < 54 mg/dl)

Comparative Effectiveness of Switching From First-Generation Basal Insulin to Glargine 300 U/ml or Degludec 100 U/ml in Type 1 Diabetes: The RESTORE-1 Study - PubMed

Comparative Effectiveness of Switching From First-Generation Basal Insulin to Glargine 300 U/ml or Degludec 100 U/ml in Type 1 Diabetes: The RESTORE-1 Study - PubMed This site needs JavaScript to work properly. Please enable it to take advantage of the complete set of features! Clipboard, Search History, and several other advanced features are temporarily unavailable. Skip to main page content An official website of the United States government Here's how you know The .gov means it’s official. Federal government websites often end in .gov or .mil. Before
sharing sensitive information, make sure you’re on a federal
government site. The site is secure. The https:// ensures that you are connecting to the
official website and that any information you provide is encrypted
and transmitted securely. Log in Show account info Close Account Logged in as: username Dashboard Publications Account settings Log out Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation Search: Search Advanced Clipboard User Guide Save Email Send to Clipboard My Bibliography Collections Citation manager Display options Display options Format Abstract PubMed PMID Save citation to file Format: Summary (text) PubMed PMID Abstract (text) CSV Create file Cancel Email citation Email address has not been verified. Go to My NCBI account settings to confirm your email and then refresh this page. To: Subject: Body: Format: Summary Summary (text) Abstract Abstract (text) MeSH and other data Send email Cancel Add to Collections Create a new collection Add to an existing collection Name your collection: Name must be less than 100 characters Choose a collection: Unable to load your collection due to an error Please try again Add Cancel Add to My Bibliography My Bibliography Unable to load your delegates due to an error Please try again Add Cancel Your saved search Name of saved search: Search terms: Test search terms Would you like email updates of new search results? Saved Search Alert Radio Buttons Yes No Email: ( change ) Frequency: Monthly Weekly Daily Which day? The first Sunday The first Monday The first Tuesday The first Wednesday The first Thursday The first Friday The first Saturday The first day The first weekday Which day? Sunday Monday Tuesday Wednesday Thursday Friday Saturday Report format

RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design - PMC

example from the treatment of Duchenne Muscular Dystrophy, in which a registry enrolling a patient who received several therapies could permit comparison of the long-term efficacies of deflazacort and prednisone [ 35 ], and better assess the efficacy of daily treatment when compared to alternative regimens [ 36 ]. In addition, long-term data collection from a large patient sample may provide important insights regarding prognostic factors, characteristics of best responders to therapies, and estimation of the duration of unsuccessful treatment after which a patient can be considered a non-responder. Another very important question that can only be assessed by long-term follow-up is the need for and the cost-effectiveness of treating pre-symptomatic patients. Even if there are few or no questions about this approach in patients with 2 or 3 SMN2 copies [ 37 ], this question will undoubtedly arise for patients with 4 copies, reflecting the creation of numerous newborn screening programs [ 38– 40 ]. The issue of treating presymptomatic patients has also been raised in late-onset Pompe disease [ 41 ] and this question should be addressed generally. Another important consideration related to treatment of asymptomatic patients, who are potential candidates for gene therapy, is the persistence of transgene expression, which can be determined over long-term follow-up of Registry patients. Review of the design of existing registries suggests that the variables evaluated in RESTORE will complement and extend analyses of existing registries, including provision of important information about the clinical course of SMA in patients receiving new treatments. Ongoing discussions with the leaders of existing SMA registries indicate that there is a large overlap in the types of data collected and a willingness, consistent with the charter of each registry, to share data for the benefit of patients, families, regulatory agencies, and clinical researchers. Given that RESTORE will provide data describing the clinical management of patients with SMA across multiple countries, there is a potential limitation due to variations in the standard of care across countries or regions, and variation in treatments based on cultural norms with the potential for missing data for some measures. This has been noted as a potential issue for all rare disease registries [ 25 ]. This is particularly important for the most severely affected patients, since they constitute the group for which the extent of medical care is the most important. In patients with less severe disease, such as those who are ambulatory, differences in standards of care are less likely to directly

InCarda Therapeutics Announces Results From the RESTORE-1 Phase 3 Trial and Plans for Continued Development - Neuro Central

for both hospital and home use Bridging Phase 1 study data to date establishes a path for future registration trials Today, InCarda, a privately held biopharmaceutical company developing an inhaled flecainide acetate inhalation solution (FlecIH-103) for the acute conversion of paroxysmal atrial fibrillation (PAF) to sinus rhythm (SR), announced significant progress made on several fronts: 1) Results of the RESTORE-1 Phase 3 trial were presented at the 2024 European Society of Cardiology meeting in London 1 on September 2, 2024, and demonstrated strong “patient proof of concept” data for the safe and effective cardioversion of patients with PAF who were administered inhaled flecainide. These results were particularly striking in light of the fact that the trial was stopped prematurely. 2) Transition to a novel and more efficient drug-delivery platform is supported by recent in vitro and Phase 1 clinical data. 3) Preliminary interim results from a Phase 1 clinical study designed to establish the dose and dosing regimen with the new drug delivery platform warrant continuation of the study for the potential advancement of FlecIH-103 into registrational trials. RESTORE-1 Phase 3 results RESTORE-1 was a randomized, placebo-controlled, Phase 3 trial, employing a commercially available jet nebulizer, to assess the efficacy and safety of FlecIH-103 for the treatment of patients with PAF. The trial was stopped prematurely after 54 of the planned 400 patients had been enrolled due to an unexpectedly low rate of blinded cardioversion compared to the experience in the Phase 2 study (INSTANT). The rate of cardioversion in RESTORE-1 was 31% in the treated group versus 47% in the Phase 2 INSTANT study. This outcome was caused by lower-than-expected flecainide plasma levels and unexpected drug-device incompatibility not previously observed in the Phase 2 clinical trial. The premature halting of the trial was not due to any safety concerns. Despite the early termination of the trial and a small sample size (n=54), an analysis showed that the 31% cardioversion rate was statistically significant in FlecIH-103-treated patients compared to placebo (p=0.04), with a median time to restoration of normal sinus rhythm (NSR) of approximately 13 minutes from the start of dosing. The study captured statistically significant pharmacoeconomic benefits from early

InCarda Therapeutics Announces Results From the RESTORE-1 Phase 3 Trial and Plans for Continued Development - Neuro Central

-treated patients compared to placebo (p=0.04), with a median time to restoration of normal sinus rhythm (NSR) of approximately 13 minutes from the start of dosing. The study captured statistically significant pharmacoeconomic benefits from early restoration of SR following administration of FlecIH-103: a) 80% of patients whose AF converted to sinus rhythm were eligible for discharge from the hospital within 2 hours of dose administration, compared to only 30% of patients whose PAF did not convert (p=0.002) b) 83% of patients who converted from PAF to NSR within 90 min of dosing were symptom-free, while only 28% of patients whose PAF did not convert to SR were symptom-free (p<0.0001) c) The need for electrical cardioversion to restore sinus rhythm was 49% in the active arm vs. 83% in the placebo arm (p<0.05). There were no safety concerns or SAEs associated with the administration of FlecIH-103 in this study and no patients discontinued the study due to an adverse event (AE) or had an AE that required treatment. In summary, these results indicate that orally inhaled flecainide can provide safe and rapid restoration of sinus rhythm in patients presenting to the emergency room with symptomatic acute PAF. We believe FlecIH-103 delivered via oral inhalation has the potential to be clinically meaningful and address the important unmet medical need for the estimated one million U.S. patients who visit ERs with PAF each year. Improved drug-delivery platform The lower-than-expected flecainide plasma levels observed in RESTORE-1 using a jet nebulizer motivated the transition to Aerogen’s novel, high-performance drug-delivery platform that enables more efficient and reliable inhaled delivery of FlecIH-103. This platform includes: a) A novel vibrating-mesh aerosol generation engine proven to deliver drugs to the deep lungs for rapid systemic absorption, provided by Aerogen Ltd., a world leader in acute care aerosol drug delivery located in Galway, Ireland, and b) A unique patient-facing breath-synchronized dosing system that helps optimize the patient’s breathing for the inhalation of FlecIH-103. The Aerogen drug-delivery platform employs the PDAP (Photo Defined Aperture

Participant- and Disease-Related Factors as Independent Predictors of Treatment Outcomes in the RESTORE-IMI 2 Clinical Trial: A Multivariable Regression Analysis - PubMed

? The first Sunday The first Monday The first Tuesday The first Wednesday The first Thursday The first Friday The first Saturday The first day The first weekday Which day? Sunday Monday Tuesday Wednesday Thursday Friday Saturday Report format: Summary Summary (text) Abstract Abstract (text) PubMed Send at most: 1 item 5 items 10 items 20 items 50 items 100 items 200 items Send even when there aren't any new results Optional text in email: Save Cancel Create a file for external citation management software Create file Cancel Your RSS Feed Name of RSS Feed: Number of items displayed: 5 10 15 20 50 100 Create RSS Cancel RSS Link Copy Actions Cite Collections Add to Collections Create a new collection Add to an existing collection Name your collection: Name must be less than 100 characters Choose a collection: Unable to load your collection due to an error Please try again Add Cancel Permalink Permalink Copy Display options Display options Format Abstract PubMed PMID Page navigation Title & authors Abstract Conflict of interest statement Figures References Associated data LinkOut - more resources Title & authors Abstract Conflict of interest statement Figures References Associated data LinkOut - more resources Open Forum Infect Dis Actions Search in PubMed Search in NLM Catalog Add to Search . 2023 May 4;10(6):ofad225. doi: 10.1093/ofid/ofad225. eCollection 2023 Jun. Participant- and Disease-Related Factors as Independent Predictors of Treatment Outcomes in the RESTORE-IMI 2 Clinical Trial: A Multivariable Regression Analysis Ignacio Martin-Loeches 1 , Andrew F Shorr 2 , Marin H Kollef 3 , Jiejun Du 4 , Maria C Losada 4 , Amanda Paschke 4 , C Andrew DeRyke 4 , Michael Wong 4 , Erin H Jensen 4 , Luke F Chen 4 Affiliations Expand Affiliations 1 Department of Intensive Care Medicine, Multidisciplinary Intensive Care Research Organization, St James's University Hospital, Trinity Centre for Health Sciences, Dublin, Ireland. 2 Section of Pulmonary, Critical Care, and Respiratory Services, MedStar Washington Hospital Center, Washington, District of Columbia, USA. 3 Division of Pulmonary and Critical Care Medicine, Department of Internal Medicine, Washington University School of Medicine, St Louis, Missouri, USA. 4 Merck & Co, Inc, Rahway, New Jersey, USA. PMID: 37383243 PMCID: PMC10297016 DOI: 10.1093/ofid/ofad225

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

, Doyle S, Guinan E, Parker A, Segurado R, et al. Patient and family co-developed participant information to improve recruitment rates, retention, and patient understanding in the rehabilitation strategies following Oesophago-gastric and Hepatopancreaticobiliary Cancer (ReStOre II) trial: protocol for a study within a trial (SWAT). HRB Open Res. 2019. Myers J, Prakash M, Froelicher V, Do D, Partington S, Atwood JE. Exercise capacity and mortality among men referred for exercise testing. N Engl J Med. 2002;346(11):793–801. Article PubMed Google Scholar Gulati M, Pandey DK, Arnsdorf MF, Lauderdale DS, Thisted RA, Wicklund RH, et al. Exercise capacity and the risk of death in women. Circulation. 2003;108(13):1554. Article PubMed Google Scholar Jones LW, Hornsby WE, Goetzinger A, Forbes LM, Sherrard EL, Quist M, et al. Prognostic significance of functional capacity and exercise behavior in patients with metastatic non-small cell lung cancer. Lung Cancer (Amsterdam, Netherlands). 2012;76(2):248–52. Article Google Scholar Moran J, Wilson F, Guinan E, McCormick P, Hussey J, Moriarty J. Role of cardiopulmonary exercise testing as a risk-assessment method in patients undergoing intra-abdominal surgery: a systematic review. Br J Anaesth. 2016;116(2):177–91. Article CAS PubMed Google Scholar Peel JB, Sui X, Matthews CE, Adams SA, Hebert JR, Hardin JW, et al. Cardiorespiratory fitness and digestive cancer mortality: findings from the aerobics center longitudinal study. Cancer Epidemiol Biomark Prev. 2009;18(4):1111–7. Article Google Scholar Peel JB, Sui X, Adams SA, Hebert JR, Hardin JW, Blair SN. A prospective study of cardiorespiratory fitness and breast cancer mortality. Med Sci Sports Exerc. 2009;41(4):742–8. Article PubMed PubMed Central Google Scholar Download references Acknowledgments The authors would like to acknowledge the assistance and support of the Wellcome Trust/HRB Clinical Research Facility at St.

Rehabilitation strategies following oesophagogastric and Hepatopancreaticobiliary cancer (ReStOre II): a protocol for a randomized controlled trial | BMC Cancer | Full Text

and social), symptom scales (fatigue, pain, nausuea and vomiting), global health status and HR-QOL scale, in addition to several single item symptom measures [ 43 ]. Cancer specific QOL issues will be assessed using the appropriate cancer subscale; QLQ-OG25 (oesophago-gastric cancer), QLQ-HCC18 (hepatocellular cancer), and QLQ-PAN26 (pancreatic cancer). To interpret the core questionnaire and cancer specific subscales, higher functional scores indicate greater functioning, whereas lower symptom scores indicate less symptom burden. Fatigue Fatigue will be measured using the Multidimensional Fatigue Inventory (MFI-20). The MFI-20 is a 20-item scale that measures the impact of fatigue in five dimensions: general, physical, cognitive, motivation and usual activities. It is scored from 0 to 20, with a cut-off score of ≥13 indicating severe fatigue. The psychometric properties of the MFI-20 have been tested and determined strong validity and reliability [ 44 ]. Qualitative data collection Qualitative methods will be utilised to investigate intervention participants’ perceptions of the impact of the ReStOre II programme on their daily lives. Data will be collected through semi-structured focus group discussions immediately post-intervention (T1) and individual interviews at 3-months follow-up (T2). Focus groups at T1 will specifically explore the impact of the ReStOre II programme on mental, physical, and social well-being, whilst also examining the value of the group-based exercise programme and education talks to recovery. Individual interviews at T2 will focus more on examining the maintenance of health behaviours acquired during participation in the ReStOre programme. Interviews and focus groups will be led by a researcher experienced in qualitative methods and audio-recorded followed by verbatim transcription in preparation for data analysis. Cost analysis Programme implementation costs will be analysed in consideration of programme costs (e.g. clinician salaries, overheads and equipment costs). Changes in HR-QOL scores will be analysed and cost-effectiveness ratios calculated. The patterns of service use and related costs will be assessed for patients in each arm of the study, and alongside the costs of the intervention a comparison will be made of the total costs and outcomes, to estimated cost-effectiveness ratios. Biosample collection Serum, plasma, and whole blood

Participant- and Disease-Related Factors as Independent Predictors of Treatment Outcomes in the RESTORE-IMI 2 Clinical Trial: A Multivariable Regression Analysis - PubMed

determine independent predictors of efficacy outcomes in the RESTORE-IMI 2 trial, to assist in treatment decision making. Methods: A stepwise multivariable regression analysis was conducted to identify variables that were independently associated with day 28 all-cause mortality (ACM), favorable clinical response at early follow-up (EFU), and favorable microbiologic response at end of treatment (EOT). The analysis accounted for the number of baseline infecting pathogens and in vitro susceptibility to randomized treatment. Results: Vasopressor use, renal impairment, bacteremia at baseline, and Acute Physiologic Assessment and Chronic Health Evaluation (APACHE) II scores ≥15 were associated with a greater risk of day 28 ACM. A favorable clinical response at EFU was associated with normal renal function, an APACHE II score <15, no vasopressor use, and no bacteremia at baseline. At EOT, a favorable microbiologic response was associated with IMI/REL treatment, normal renal function, no vasopressor use, nonventilated pneumonia at baseline, intensive care unit admission at randomization, monomicrobial infections at baseline, and absence of Acinetobacter calcoaceticus-baumannii complex at baseline. These factors remained significant after accounting for polymicrobial infection and in vitro susceptibility to assigned treatment. Conclusions: This analysis, which accounted for baseline pathogen susceptibility, validated well-recognized patient- and disease-related factors as independent predictors of clinical outcomes. These results lend further support to the noninferiority of IMI/REL to piperacillin/tazobactam and suggests that pathogen eradication may be more likely with IMI/REL. Clinical trials registration: NCT02493764 . Keywords: HABP; VABP; antibiotic; imipenem; relebactam. © The Author(s) 2023. Published by Oxford University Press on behalf of Infectious Diseases Society of America. PubMed Disclaimer Conflict of interest statement Potential conflicts of interest. L. F. C., J. D., M. C. L., A. P., C. A. D., M. W., and E. H. J. are employees of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co, Inc, Rahway, New Jersey, who may own stock and/or hold stock options in the Mer

RESTORE-IMI 1: A Multicenter, Randomized, Double-blind Trial Comparing Efficacy and Safety of Imipenem/Relebactam vs Colistin Plus Imipenem in Patients With Imipenem-nonsusceptible Bacterial Infections - PubMed

RESTORE-IMI 1: A Multicenter, Randomized, Double-blind Trial Comparing Efficacy and Safety of Imipenem/Relebactam vs Colistin Plus Imipenem in Patients With Imipenem-nonsusceptible Bacterial Infections - PubMed This site needs JavaScript to work properly. Please enable it to take advantage of the complete set of features! Clipboard, Search History, and several other advanced features are temporarily unavailable. Skip to main page content An official website of the United States government Here's how you know The .gov means it’s official. Federal government websites often end in .gov or .mil. Before
sharing sensitive information, make sure you’re on a federal
government site. The site is secure. The https:// ensures that you are connecting to the
official website and that any information you provide is encrypted
and transmitted securely. Log in Show account info Close Account Logged in as: username Dashboard Publications Account settings Log out Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation Search: Search Advanced Clipboard User Guide Save Email Send to Clipboard My Bibliography Collections Citation manager Display options Display options Format Abstract PubMed PMID Save citation to file Format: Summary (text) PubMed PMID Abstract (text) CSV Create file Cancel Email citation Email address has not been verified. Go to My NCBI account settings to confirm your email and then refresh this page. To: Subject: Body: Format: Summary Summary (text) Abstract Abstract (text) MeSH and other data Send email Cancel Add to Collections Create a new collection Add to an existing collection Name your collection: Name must be less than 100 characters Choose a collection: Unable to load your collection due to an error Please try again Add Cancel Add to My Bibliography My Bibliography Unable to load your delegates due to an error Please try again Add Cancel Your saved search Name of saved search: Search terms: Test search terms Would you like email updates of new search results? Saved Search Alert Radio Buttons Yes No Email: ( change ) Frequency: Monthly Weekly Daily Which day? The first Sunday The first Monday The first Tuesday The first Wednesday The first Thursday The first Friday The